Actively Recruiting
Lentiviral Gene Therapy for Chronic Granulomatous Disease (CGD)
Led by Shenzhen Geno-Immune Medical Institute · Updated on 2026-04-24
10
Participants Needed
1
Research Sites
78 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating a gene therapy treatment for Chronic Granulomatous Disease (CGD), a rare inherited disorder that causes severe infections due to defects in the NADPH oxidase enzyme complex. This trial aims to assess the safety and effectiveness of a self-inactivating lentiviral vector called TYF-CGD, which is designed to correct the defective gene causing CGD by restoring immune function and reducing infection frequency. The treatment involves infusing patients with their own hematopoietic stem cells that have been modified outside the body using the TYF lentiviral vector to carry a functional gene. The dose ranges from 1 to 10 million gene-modified cells per kilogram of body weight. This advanced gene transfer protocol is being studied to observe immune recovery and long-term correction of immune dysfunction. Participants will be monitored over a long period, with a 15-year follow-up to assess overall survival and gene marking in bone marrow cells. Additional evaluations during the first year will include infection frequency, immune function recovery, clinical exams, and laboratory tests. The study involves close safety monitoring and long-term observation to understand the treatment's impact on immune system restoration and infection control.
CONDITIONS
Brief Title
Lentiviral Gene Therapy for CGD
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosed with Chronic Granulomatous Disease of any age
- Confirmed molecular diagnosis by DNA sequencing and lab evidence of reduced NADPH-oxidase activity
- Karnofsky-Index of 70% or higher
- History of at least one severe infection or inflammatory complication requiring hospitalization despite medication
- Provided written informed consent or assent if age 7 or older
You will not qualify if you...
- Contraindications for leukapheresis such as anemia (Hb <8g/dl), cardiovascular instability, or severe coagulopathy
- Contraindications for conditioning medication
- Female patients who are pregnant or breastfeeding as determined by history or pregnancy test
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single treatment event with ongoing monitoring
Participants receive an infusion of lentiviral TYF-CGD-modified autologous stem cells to correct their immune dysfunction.
1 infusion visit and frequent follow-up visits during initial recovery
Duration - Up to 15 years
Participants are monitored for safety, immune function recovery, and infection frequency for up to 15 years after treatment.
Regular visits during the first year followed by long-term annual visits
Trial Site Locations
Total: 1 location
1
Shenzhen Geno-immune Medical Institute
Shenzhen, Guangdong, China, China
Actively Recruiting
Research Team
L
Lung-Ji Chang, Ph.D
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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